An International Observational Registry Study to Further Describe Long-term Safety and Effectiveness of Palovarotene in Patients With Fibrodysplasia Ossificans Progressiva (FOP)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Ipsen
- 入组人数
- 100
- 试验地点
- 8
- 主要终点
- Percentage of Participants With Serious and Non-serious treatment-related TEAEs
研究概览
简要总结
The participants in this registry study will have fibrodysplasia ossificans progressiva (FOP).
FOP is an ultra-rare, severely disabling disease characterized by new bone formation in areas of the body where bone is not normally present (heterotopic ossification (HO)).
HO is often preceded by painful, recurrent episodes of soft tissue swelling (flare-ups).
This registry study will take place in countries where the treatment, known as palovarotene, has been approved for use. Participants will either be treated with palovarotene (i.e already be receiving palovarotene as prescribed by their treating physician according to locally approved product information) or untreated with palovarotene.
The main aim of this registry study will be to collect and assess real-world safety data on children and adult participants with FOP treated with palovarotene.
This registry study will also describe the effectiveness of palovarotene in exposed participants, including the effect on everyday activities and physical performance.
In addition, this registry study aims to descriptively compare key safety outcomes (i.e. flare-up episodes, growth outcomes, and bone fractures) between participants exposed and unexposed to palovarotene.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 8 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult or child with FOP who have been prescribed palovarotene (prior to and independently of the decision to enroll the patient in this registry study and as per local label) by their treating physician according to the locally approved product information;
- •Signed informed consent as per local regulations must be obtained and maintained. Consent/assent from the participant should be obtained as appropriate before any registry study data collection are conducted. If applicable, parents or legally authorized representatives must give signed informed consent.
排除标准
- •Currently participating in a palovarotene clinical trial;
- •Currently participating in any interventional clinical trial for FOP;
- •Have any contraindication to palovarotene as per the locally approved label (except for pregnant women who have previously received and discontinued palovarotene at any time during the pregnancy and who will be included for safety follow-up).
研究组 & 干预措施
Exposed Cohort
Participants treated with palovarotene (i.e already be receiving palovarotene as prescribed by their treating physician according to locally approved product information)
Unexposed Cohort
Participants untreated with palovarotene
结局指标
主要结局
Percentage of Participants With Serious and Non-serious treatment-related TEAEs
时间窗: From Baseline up to 30 days after the last palovarotene dose.
Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
Percentage of Participants With all serious TEAEs, whether or not they are considered as related to the palovarotene
时间窗: From Baseline up to 30 days after the last palovarotene dose.
Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs), whether or not they are considered as related to palovarotene
时间窗: From Baseline up to 30 days after the last palovarotene dose.
Adverse events will be coded according to MedDRA and will be classified by Preferred Term (PT) and system organ class (SOC).
Percentage of Participants With nonserious TEAEs whether or not they are considered as related to the palovarotene.
时间窗: From Baseline up to 30 days after the last palovarotene dose.
Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
次要结局
- Change from Baseline in Cumulative Analogue Joint Involvement Scale (CAJIS) total score(From Baseline and every six months up to eleven years.)
- Change from Baseline in use of assistive devices(From Baseline and every six months up to eleven years.)
- Change from Baseline in percent of worst score for Fibrodysplasia Ossificans Progressiva-Physical Function Questionnaire (FOP-PFQ) total score(From Baseline and every six months up to eleven years.)
- Change from Baseline in observed and percent predicted Forced Vital Capacity (FVC)(From Baseline and every six months up to eleven years.)
- Change from Baseline in observed and percent predicted Forced Expiratory Volume in one second (FEV1)(From Baseline and every six months up to eleven years.)
- Change from Baseline in absolute and percent predicted FEV1/FVC ratio(From Baseline and every six months up to eleven years.)
- Change from Baseline in observed and percent predicted Diffusion Capacity of the Lung for Carbon Monoxide (DLCO)(From Baseline and every six months up to eleven years.)
- Change from Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) physical and mental function (mean global physical and mental health score converted into T-scores) for participants ≥15 years old(From Baseline and every six months up to eleven years.)
- Change from Baseline in PROMIS overall quality of life (QoL) (mean total score converted into T-scores) for participants <15 years old(From Baseline and every six months up to eleven years.)
- Change from Baseline in annualized number of Investigator-reported flare-ups by body location and overall(From Baseline and every six months up to eleven years.)
- Number of flare-up outcomes(From Baseline and every six months up to eleven years.)
- Flare-up Duration(From Baseline and every six months up to eleven years.)
- Number of flare-ups(From 12 months before inclusion Baseline up to eleven years.)
- Mean difference between chronological age and bone age(From Baseline and every six months up to eleven years.)
- Change from Baseline in Cumulative Analogue Joint Involvement Scale (CAJIS) total score(From Baseline and every six months up to eleven years.)
- Change from Baseline in use of assistive devices(From Baseline and every six months up to eleven years.)
- Change from Baseline in percent of worst score for Fibrodysplasia Ossificans Progressiva-Physical Function Questionnaire (FOP-PFQ) total score(From Baseline and every six months up to eleven years.)
- Change from Baseline in observed and percent predicted Forced Vital Capacity (FVC)(From Baseline and every six months up to eleven years.)
- Change from Baseline in observed and percent predicted Diffusion Capacity of the Lung for Carbon Monoxide (DLCO)(From Baseline and every six months up to eleven years.)
- Change from Baseline in observed and percent predicted Forced Expiratory Volume in one second (FEV1)(From Baseline and every six months up to eleven years.)
- Change from Baseline in absolute and percent predicted FEV1/FVC ratio(From Baseline and every six months up to eleven years.)
- Change from Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) physical and mental function (mean global physical and mental health score converted into T-scores) for participants ≥15 years old(From Baseline and every six months up to eleven years.)
- Change from Baseline in PROMIS overall quality of life (QoL) (mean total score converted into T-scores) for participants <15 years old(From Baseline and every six months up to eleven years.)
- Change from Baseline in annualized number of Investigator-reported flare-ups by body location and overall(From Baseline and every six months up to eleven years.)
- Number of flare-up outcomes(From Baseline and every six months up to eleven years.)
- Flare-up Duration(From Baseline and every six months up to eleven years.)
- Number of flare-ups(From 12 months before inclusion Baseline up to eleven years.)
- Number of locations impacted per participant(From Baseline and annually up to eleven years.)
- Movement mobility change(From Baseline and annually up to eleven years.)
- Evolution of impacted location (how many times a location is impacted) per location(From Baseline and annually up to eleven years.)
- Movement mobility outcomes: Percentage of participants with new bone growth overall and by flare-up status(From Baseline and every six months up to eleven years.)
- Percentage of participants with any fractures overall(From baseline up to 30 days after the last palovarotene dose.)
- Change from Baseline in annualized number of fractures by body location and overall(From baseline up to 30 days after the last palovarotene dose.)
- Incidence of pregnancy(From Baseline and every month up to 30 days after the last palovarotene dose.)
- Description of pregnancy characteristics and outcomes(From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.)
- Description of pregnancy related Adverse Events (AEs)(From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.)
- Description of birth and developmental outcomes through the first year after birth(From Baseline and every six months up to eleven years.)
- Change from Baseline in height z-score(From Baseline and every six months up to eleven years.)
- Mean difference between chronological age and bone age(From Baseline and every six months up to eleven years.)
- Frequency of premature physeal closure (PPC) overall(From baseline up to 30 days after the last palovarotene dose)
- Mean dose/year of palovarotene for participants with FOP exposed and unexposed to palovarotene for chronic treatment(From Baseline and every six months up to eleven years.)
- Mean dose/cycle of palovarotene for participants with FOP exposed and unexposed to palovarotene for flare-up treatment(From Baseline and every six months up to eleven years.)
- Description of birth and developmental outcomes through the first year after birth(From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.)